Shares of Sangamo Therapeutics (NASDAQ:SGMO) rose over 16% today after the company announced that the first patient had been dosed in a phase 1/2 trial evaluating the potential for its gene-editing technology to treat Hunter syndrome. It's the first time doctors in the United States have attempted to use gene editing in a live patient in a controlled setting, so investors are understandably happy to reach the milestone.
Sangamo Therapeutics uses a gene-editing tool called zinc finger nucleases (ZFN), which hasn't received as much press in the last two years as CRISPR gene editing. That's partially due to how tightly the company has controlled the technology, whereas multiple companies have snagged licenses to CRISPR tools. Nevertheless, ZFN just beat CRISPR to the clinic. The latter isn't expected to reach that milestone until mid-2018 when Editas Medicine begins its first phase 1 trial.
As of 3:29 p.m. EST, the stock had settled to a 13.6% gain.
Aside from bragging rights, it doesn't matter much whether ZFN or CRISPR reaches the clinical milestone first. They aren't competing head-to-head for too many indications at the moment. In fact, the more gene-editing therapies that reach the clinic, the better, as it could help to set a new precedent for the general approach of curing and treating diseases by "precisely" altering a patient's DNA.
Of course, given that the trial is still in the earliest stages of development -- dosing the first human patient in a clinical setting -- today's move is a little more ceremonious than anything else. Investors will have more important updates as the trial progresses. If patients with rare metabolic disorders that have no other treatment options respond well, or even cured, by gene-editing therapies, then it could be a big boon for Sangamo Therapeutics' future prospects.
Sangamo Therapeutics stock has been on a tear in the last 12 months. Investors have clearly regained their confidence in the company thanks to a new approach in the clinic, which focuses more on gene editing than past gene therapies using ZFNs. Given that the phase 1/2 trial in Hunter syndrome is one of three early-stage trials using the new gene-editing tools developed by the company, investors may expect a few more catalysts in the near term. Whether or not the stock increases are sustainable is up for debate.